17 Jun 2002
Scientists in Ohio said Monday they had made a synthetic miniature human chromosome, which could generate fresh approaches to the difficult field of gene therapy.
In the April edition of the journal Nature Genetics, a team from Case Western Reserve University in Cleveland and Athersys Inc. report that they were able to create asynthetic ''microchromosome'' out of elements found naturally in the human body. It is about one-fifth to one-tenth the size of a real human chromosome andcontains less genetic material.
Gene therapy requires introducing a specific gene to target cells to fix a defect, such as cystic fibrosis or a form of muscular dystrophy. Most attempts so far haveinvolved using an altered virus to carry the gene to the site, with little success. These viruses can sometimes produce an undesired immune response or the genecannot produce consistently. The hope is that a synthetic mini-human chromosome could eventually be used to shuttle the desired gene to the site needing repair
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